转基因皮肤挽救了一个危在旦夕的男孩,双语阅

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Genetically Altered Skin Saves A Boy Dying Of A Rare Disease-转基因皮肤挽救了一个危在旦夕的男孩

In the future, we may not need to rely on human donations for life-saving skin grafts.

November 8, 20171:28 PM ET

未来,可挽救生命的皮肤移植或许无需再依赖人类自身捐献了。

Heard onAll Things Considered

That’s the goal of XenoTherapeutics, a Boston-based biotech nonprofit. Last week, the US Food and Drug Administration approved the group’s initial application for temporary skin grafts curated from genetically modified pigs. This means that they can start testing pig skin grafts on people who have experienced severe burns. It’s the first time that an animal organ has been cleared for human testing in the US.

Richard Harris

这就是总部设在波士顿的非营利性生物科技企业塞诺移植治疗公司的目标。上周,美国食品和药物管理局批准了该公司使用基因改良猪的皮作为临时移植皮肤的初步申请。这意味着,他们可以开始对严重烧伤者进行猪皮移植测试。这也是美国首次批准用动物器官进行人体测试。

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Skin, the body’s largest organ, plays a crucial role in the immune system by blocking pathogens from reaching our vulnerable internal organs. It also holds in water, electrolytes, and other nutrients, and helps the body maintain a constant temperature. People with severe skin damage are at a high risk of developing deadly infections or organ failure as a result of changes in temperature or hydration.

Researchers

皮肤是人体最大的器官,可阻止病原体侵入人体脆弱的体内器官,因此在免疫系统中发挥着至关重要的作用。此外,皮肤可锁住水分、电解质及其他营养物质,还可帮助人体保持体温稳定。皮肤严重受损者因体温或水合状态的改变而患致命性感染或器官衰竭的风险很高。

grew sheets of genetically altered skin cells in the lab and used them

Skin grafts can help protect these patients as they heal. At the moment, the only skin grafts available in the US come from cadavers who have agreed to be organ donors, or patients who have undergone surgery to remove excess skin after dramatic weight loss. These human skin used for grafts are a “rare commodity.”

to treat a boy withlife-threatening epidermolysis bullosa.

移植皮肤可在患者痊愈过程中对患者加以保护。当前,在美国获取移植皮肤的唯一途径是使用同意捐献器官者死后留下的皮肤,或因体重严重下降做多余皮肤去除术的患者皮肤。用于移植的人类皮肤可谓是“稀缺商品”。

研究人员在实验室中中培养转基因皮肤细胞,用它们来治得了疗危及生命的大疱性表皮松解症的男孩。

XenoTherapeutics, which gets its name from xenotransplantation, or animal-to-human transplants, has bred pigs that have skin remarkably similar to our own. Although pig skin normally produces a type of sugar human skin does not, these pigs have been genetically modified not to make it. Grafts from these pigs are therefore more likely to slide under the radar of the host’s immune system—at least temporarily. The idea is that they could be used for immediate burn treatment, followed human skin graft treatment later.

CMR Unimore/Nature

塞诺移植治疗公司的英语名就源自异种器官移植概念——即把动物器官移植给人体。该公司饲养的猪,其猪皮与我们人类的皮肤极为相似。猪皮一般会生成一种糖而人皮则不会,为了使这些猪不生成这种糖,该公司对这些猪实施了基因改良。这样一来,这些猪皮通过接受移植者免疫系统监测的可能性就更高了——至少短期内可以通过。这些猪皮可被用于紧急烧伤处理,之后再进行人皮移植。

A

“I would venture that if we did a Coke and Pepsi side-by-side comparison… you’d be hard-pressed to tell which was the human cadaveric allograft versus [the pig graft],” said XenoTherapeutics CEO Paul Holzer.

child who was on the verge of death from a rare inherited disease has

塞诺移植治疗公司首席执行官保罗·霍尔泽说:“我敢说,这就像你把可口可乐和百事可乐放在一起进行对比,你很难分得清哪个是源自人类尸体的同种异体移植皮肤,哪个是移植猪皮。”

been treated with genetically engineered skin cells that replaced most

The nonprofit has been working closely with physicians at Massachusetts General Hospital, who will help conduct the clinical trials starting next month. The first trial will only be testing the grafts’ safety and tolerability in six patients with severe burns. Assuming the results are positive after a month, the grafts will need to undergo two more stages of testing before they can be approved for widespread clinical use.

of the skin on his body.

这家非营利性企业一直在与马萨诸塞综合医院的医生们进行紧密合作,这所医院将从下月起协助进行临床实验。首次临床实验将只在6名严重烧伤者身上测试移植的安全性以及耐受性。假设一个月后临床实验结果是积极的,移植工作还要进行两个阶段的测试,而后才可获批推广到临床使用。

一个因罕见遗传疾病濒临死亡的孩子正在用转基因皮肤细胞替代了自身大部分的皮肤。

Several other groups around the world are working to make animal organs suitable for clinical medicine. In Brazil, researchers are exploring using tilapia skin for use as temporary bandages for burn victims whose skin is regrowing. Just last week, scientists from Germany reported that they had made a crucial step in keeping baboons given genetically modified pig hearts alive for half a year. Their success suggests that pig hearts could one day be used to treat patients with heart failure.

The

除塞诺移植治疗公司外,目前全球还有多个团体在研究将动物器官适用于临床医学。例如在巴西,研究人员正在研究用罗非鱼的鱼皮作为正在重新长出皮肤的烧伤者的临时绑带。而就在上周,多位德国科学家报告称他们也迈出重要一步,成功地让接受基因改良猪心脏移植的狒狒存活了半年时间。这表明猪心脏未来将可能被用来治疗心衰患者。

treatment represents a notable success for the field of genetherapy,

which has suffered manysetbacks. And it's potentially good news for

children suffering from a painful and often deadly skin condition called

epidermolysis bullosa.

虽然经历了许多挫折,但该疗法在基因治疗领域还是取得了显著的成功,对于患有大疱性表皮松解症这种疼痛且经常致命皮肤病的儿童来说,这可能是个好消息。

In

this disease, children are born with a flawed gene that prevents the

outer layer of the skin, theepidermis, from binding to the inner layer.

This can cause excruciatingblistersto form all over these children's

bodies.

在这种疾病中,儿童天生就患有一个基因缺陷,他阻止皮肤、表皮的外层与内层结合。这会在儿童的全身引起水泡。

In

the case in Europe, a 7-year old boy ended up in the hospital back in

2015 after 60 percent of his epidermis had sloughed off. Tobias

Rothoeft, a surgeon at a burn unit at Ruhr University in Bochum,

Germany, says he and his colleagues tried everything — including a skin

transplant from the boy's father —to no avail.

在欧洲的一个病例,一个7岁的男孩住进了医院,2015年后他的60%的皮肤已经脱落。Tobias Rothoeft,在波鸿的鲁尔大学的一名烧伤科医生德国说,他和他的同事们尝试了一切——包括男孩父亲的皮肤移植——但都无济于事。

"After nearly two months we were absolutely sure there was nothing we could do for this kid and that he would die," Rothoeft said in a telephone news conference hosted byNature, which publishedthe studyonline Wednesday.

“在经过近两个月我们绝对相信我们帮不了这个孩子,他会死的,”Rothoeft在《自然》杂志的电话新闻发布会上说,这段话在周三也被发布到了网络上。

Rothoeft and his colleagues took one last look around the medical literature and learned of researchers in Italy who were experimenting with anew treatment00126-4)for this disease. Michele De Luca and colleagues at the University of Modena and Reggio Emilia were genetically engineering skin cells to repair the inborn flaw.

Rothoeft和他的同事们偶然看到了一份医学文献,得知意大利的研究人员正在试验治疗这种疾病的新的治疗方法。来自意大利摩德纳大学Michele De Luca和科尔正利用基因工程的皮肤细胞修复基因的先天缺陷。

De

Luca used a virus toinserta healthy gene into cells taken from the

boy's skin. Some of those cells,stem cells,multiply indefinitely. So De

Luca was able to grow entire sheets of engineered epidermis, which were

shipped to the hospital in Germany.

De Luca用一种病毒将健康的基因插入从男孩皮肤中取出的细胞中。其中一些细胞,干细胞,无限期繁殖。所以De Luca能够一整面的转基因的人工表皮,它被运到了德国的医院。

De

Luca had used this procedure successfully in 2006 to replace a

relatively small patch of skin on another patient. But this boy needed

to have 80 percent of his skin replaced with grafts of this genetically

modified material. It took two operations, both in the fall of 2015.

De Luca在2006年成功地使用了这个办法来替换另一个病人的一小块皮肤。但是这个男孩需要80%的皮肤移植。在2015年秋季,他一共进行了两次手术。

"In

the first one, we grafted all four limbs," De Luca told reporters in

the press call. "In the second operation we grafted the remaining part

of the body, mainly the back."

De Luca在接受记者采访时对记者说:“第一次,我们移植了所有四肢的皮肤。”在第二次手术中,我们移植了身体的剩余部分,主要是背部。

After

eight months in the intensive care unit, the boy was well enough to go

home. And, two years later, he is in school, even playing soccer.

在ICU呆了八个月后,这个男孩已经康复了,他可以回家了。两年后,他上学了,甚至可以踢足球。

"The

kid is doing quite well," Rothoeft said. "The skin is of good quality,

it doesn't need any ointments or stuff like that. It's perfectly smooth

and it is quite stable. And if he gets any bruises, they

justheallikebruisesin every other kid."

“这个孩子回复得很好,”Rothoeft说。“皮肤的质量是好的,它不需要任何药膏或类似的东西。它非常平滑,而且相当稳定。如果他有瘀伤,他们就像其他孩子的瘀伤一样会自己愈合。”

Onelingeringquestion is the concern that gene therapies like this, involving viruses, can increase the risk of cancer. That's because the viruses insert the new gene randomly into human DNA. Anattemptat using gene therapy to treat severe combined immuneodeficiency (SCID) in 2002 ended up triggering cancer in some patients.

一个挥之不去的问题是,这种基因疗法,包括病毒,可能会增加患癌症的风险。这是因为病毒将新基因随机地插入人类的DNA中。2002年,在一次尝试用基因疗法治疗重症联合性免疫缺陷(SCID)的时候,所引发的癌症让一名换着住进了医院。

That bad result set back the field of gene therapy, though there have since been successes treatingSCID, and most recently, cancer.

这一不良结果阻碍了基因治疗领域的发展,尽管后来已经成功地治疗了SCID和最近的癌症。

"Certainly

it is a potential problem," De Luca said. But he generated hundreds of

millions of cells during this procedure and didn't see anything of

concern. And in this case, clearly the benefits of treating the boy

outweighed the risks.

“当然,这是一个潜在的问题,”De Luca说。但他在这个过程中产生了上亿的皮肤细胞,并没有发生任何令人担忧的事情。在这种情况下,显然治疗男孩的好处是远远大于风险的。

News of this is just starting to trickle out to advocates who have children with epidermolysis bullosa.

这一消息就是被一名患有大疱性表皮松解症的孩子的父母发布的。

"I think it's groundbreaking," says Brett Kopelan, who heads a U.S. organization focused on this disease, known by its acronym,debra. "I think it's incredibly exciting."

“我认为这是开创性的,”关注这个疾病美国协会的负责人,Brett Kopelan说,“我认为这非常令人兴奋。”

His 10-year-old daughter Rafi has a severe case.

他10岁的女儿Rafi患有这种疾病。

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Rafi

Kopelan, 10, has epidermolysis bullosa, which causes painful blisters

of the skin and mucous membranes. Her father, Brett, is at right.

Rafi Kopelan,10岁,患有大疱性表皮松解症,导致痛苦的水泡性皮肤和粘膜。她的父亲,布雷特,在右边。

Courtesy of Brett Kopelan

"Imagine

the last time you had a paper cut and you put some Purell on it and it

stung, right?" Kopelan says. "Now imagine that being 60 percent of your

body.

“想象一下,你上一次被纸割到,你用一些消毒液来清洗,慢慢有点刺痛,对吗?”Kopelan说。”现在想象一下,如果你身体的60%的皮肤都经历这种情况的时候。

"When

you have to do a bath and bandage change every day, you are subjected

to severe torture. It's incredibly painful, and it can take up to 3 to 4

hours a day," he says. "As a parent, there's not a day that goes by

that a little bit of my heart doesn't break.

当你每天必须洗澡和换绷带时,你会遭受严重的折磨。这是令人难以置信的痛苦,每天,它可能需要长达3至4小时,”他说。作为父母,每时每刻,我都是心碎不已。

"Yet

Rafi, knowing that it's going to be an incredibly painful couple of

hours, walks into the bath and bandage room ... so that we can clean her

wounds to make sure they don't get infected and to prevent a

potentially life-threatening situation.

然而,Rafi,知道这将是痛苦的几小时,走进浴室和绷带的房间…,我们可以清理她的伤口,避免感染和预防可能危及生命的情况。

"She's the bravest person I know."

“她是个勇敢的姑娘”

In

addition to the painfulblisters, Rafi needs frequent throat surgeries,

because her condition also affects mucous membranes. She often uses a

wheelchair because it's so painful to walk.

除了痛苦的水泡,Rafi需要频繁喉咙手术,因为她的病情也影响粘膜。她经常坐轮椅,因为走路太痛苦了。

"My

daughter would love to be able to not have to wear bandages on a daily

basis, she'd love to jump in a pool without worrying about it hurting,

or taking a shower — or even wearing shoes."

“我女儿很想每天都可以不用带绷带,她喜欢在游泳池里跳,不用担心会受伤,或者洗澡,甚至穿鞋。”

The skin therapy described in theNaturepaper wouldn't cure her — in fact, it targets a different genetic defect that causes the same condition. But a similar approach could reduce the agony of daily living. And Kopelan says medical interest in this disease is now growing rapidly.

《自然》描述的皮肤疗法无法真正治愈她——事实上,它针对的是一种导致相同情况的不同的遗传缺陷。但是类似的方法可以减少每天生活上的痛苦。Kopelan说,这种病目前正迅速引起医学界的兴趣。

"We've

gone from zero biotechnology and pharmaceutical [companies] to like 12

companies, so we're really at aninflectionpoint right now," he says.

“我们已经从0家生物技术和制药[公司]发展到了12家公司,所以我们现在正处在一个拐点,”他说。

Experimentaltreatments are getting under way in the United States and Asia, as well as in Europe. Peter Marinkovich, Jean Tang and colleagues at the Stanford University School of Medicine areusing the same approachas De Luca, and they have treated seven children using smaller patches of skin.

美国和亚洲以及欧洲正在进行实验性治疗。Peter Marinkovich,Jean Tang和他的同事们在斯坦福大学医学院使用和De Luca一样的方式,用小块皮肤治疗了七个孩子。

Marinkovich

365游戏官方网站,tells Shots that their long-term goal is to treat a child's entire

body, and the research is gradually laying the groundwork to do that.

The severely injured child in Germany offered a unique opportunity to

try that, and the encouraging results are generating more enthusiasm. "I

was super impressed when I saw [the] results," Marinkovich says.

Marinkovich告诉记者,他们的长期目标是治疗孩子的整个身体,和以逐渐的研究做铺垫。德国受重伤的一些儿童提供了一个尝试的机会,令人鼓舞的结果会激发更多的热情。当我看到结果后给我留下了超级深刻的印象,”Marinkovich说。

“本译文仅供个人研习、欣赏语言之用,谢绝任何转载及用于任何商业用途。本译文所涉法律后果均由本人承担。本人同意简书平台在接获有关著作权人的通知后,删除文章。”

转基因皮肤挽救了一个危在旦夕的男孩,双语阅读。原文来自  NPRRichard HarrisNovember 8, 20171:28 PM ET

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